Department of Pediatrics, University of Utah, Salt Lake City, Utah, USA.
Department of Pharmacy, Children's Hospital Colorado, University of Colorado Anschutz Medical Campus, Aurora, Colorado, USA.
Pediatr Blood Cancer. 2023 Mar;70(3):e30190. doi: 10.1002/pbc.30190. Epub 2023 Jan 5.
The event-free survival of pediatric low-grade gliomas is poor, and patients often require multiple treatment strategies. While MEK and RAF inhibitors are efficacious in early-phase trials, not all patients respond, and many experience progression following completion of therapy. Evaluating combination therapies that may enhance efficacy or prolong disease stabilization is warranted. We report our institutional experience using concurrent trametinib and lenalidomide in the treatment of primary pediatric central and peripheral nervous system tumors. Two of four patients using this combination therapy experienced severe thromboembolic events, necessitating discontinuation of therapy. This combination requires further investigation, and we urge caution if used.
儿科低级别胶质瘤的无事件生存情况较差,患者通常需要多种治疗策略。虽然 MEK 和 RAF 抑制剂在早期试验中有效,但并非所有患者都有反应,许多患者在完成治疗后会出现进展。评估可能增强疗效或延长疾病稳定的联合治疗是有必要的。我们报告了我们机构使用同时使用曲美替尼和来那度胺治疗原发性儿童中枢和外周神经系统肿瘤的经验。在使用这种联合疗法的 4 名患者中有 2 名出现严重的血栓栓塞事件,需要停止治疗。这种联合疗法需要进一步研究,如果使用,我们敦促谨慎。